Temozolomide is an active agent in children with recurrent medulloblastoma/primitive neuroectodermal tumor: An Italian multi-institutional phase II trial

Graziella Cefalo, Maura Massimino, Antonio Ruggiero, Giuseppe Barone, Vita Ridola, Filippo Spreafico, Paolo Potepan, Massimo E. Abate, Maurizio Mascarin, Maria Luisa Garrè, Giorgio Perilongo, Enrico Madon, Cesare Colosimo, Riccardo Riccardi

Research output: Contribution to journalArticlepeer-review

Abstract

BackgroundThe aim of this study was to assess the objective response rate (ORR) of children and young adults with recurrent medulloblastoma/primitive neuroectodermal tumor (MB/PNET) treated with temozolomide (TMZ). The secondary purpose was to analyze the toxicity profile of TMZ when administered orally for 5 days in 3 divided daily doses every 28 days.MethodsForty-two patients with recurrent MB/PNET, aged 21 years and younger, were recruited. Patients were treated with oral TMZ. Starting doses ranged from 120 to 200 mg/m 2/day based on previous treatments. A craniospinal MRI was performed prior to the first cycle of TMZ and following every 2 cycles of treatment.ResultsMedian age was 10 years (range, 2-21 years). Forty of 42 patients were assessed for response and toxicity. The objective response rate was 42.5%: 6 patients achieved a complete response, 11 had a partial response, and 10 had stable disease. Progression-free survival rates for all patients at 6 and 12 months were 30% and 7.5%, respectively. Their median overall survival rates at 6 and 12 months were 42.5% and 17.5%, respectively. No major extrahematological effects or life-threatening events were reported. The most common grade 3/4 toxicity included thrombocytopenia (17.5%), neutropenia (7.5%), and anemia (2.5%).ConclusionsTMZ proved to be an effective agent in children and young adults with MB/PNET, heavily pre-treated, with a tolerable toxicity profile.

Original languageEnglish
Pages (from-to)748-753
Number of pages6
JournalNeuro-Oncology
Volume16
Issue number5
DOIs
Publication statusPublished - 2014

Keywords

  • Medulloblastoma
  • Phase II trial
  • Temozolomide

ASJC Scopus subject areas

  • Cancer Research
  • Oncology
  • Clinical Neurology
  • Medicine(all)

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